Gaucher disease, type 1 |
Acid beta-glucosidase deficiency; GBA deficiency; Non-Neuronopathic Gaucher Disease |
Gaucher's disease (pronounced goʃeɪ) is the most common of the lipid storage diseases. It is caused by a deficiency of the enzyme glucocerebrosidase, leading to an accumulation of its substrate, the fatty substance glucocerebroside. Fatty material can collect in the spleen, liver, kidneys, lungs, brain and bone marrow. Symptoms may include enlarged spleen and liver, liver malfunction, skeletal disorders and bone lesions that may cause pain, severe neurologic complications, swelling of lym ...
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Gaucher disease, type 1 Clinical Trials
- A Study of the Efficacy and Safety of Genz-112638 in Type 1 Gaucher Patients
ClinicalTrials.gov Identifier: NCT00358150 - Active, not recruiting
Gaucher Disease, Type 1; Cerebroside Lipidosis Syndrome; Glucocerebrosidase Deficiency Disease; Glucosylceramide Beta-Glucosidase Deficiency Disease; Gaucher Disease, Non-Neuronopathic Form
- Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
ClinicalTrials.gov Identifier: NCT00364858 - Active, not recruiting
Gaucher Disease, Type 1; Cerebroside Lipidosis Syndrome; Glucocerebrosidase Deficiency Disease; Glucosylceramide Beta-Glucosidase Deficiency Disease; Gaucher Disease, Non-Neuronopathic Form
- Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
ClinicalTrials.gov Identifier: NCT00391625 - Active, not recruiting
Gaucher Disease, Type 1
- Safety Study of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
ClinicalTrials.gov Identifier: NCT00433147 - Recruiting
Gaucher Disease, Type 1
- Multicenter Open-Label Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
ClinicalTrials.gov Identifier: NCT00478647 - Recruiting
Gaucher Disease, Type 1
- Oral Miglustat in Adult Patients With Stable Type 1 Gaucher Disease
ClinicalTrials.gov Identifier: NCT00319046 - Recruiting
Type 1 Gaucher Disease
- A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
ClinicalTrials.gov Identifier: NCT00365131 - Completed
Gaucher Disease Type I; Cerebroside Lipidosis Syndrome; Clucocerebrosidase Deficiency Disease; Glucosylceramide Beta-Glucosidase Deficiency Disease; Gaucher Disease, Non-Neuronopathic Form
- A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease
ClinicalTrials.gov Identifier: NCT00553631 - Not yet recruiting
Gaucher Disease, Type 1
- A Study of GA-GCB Enzyme Replacement Therapy in Gaucher Disease
ClinicalTrials.gov Identifier: NCT00430625 - Recruiting
Gaucher Disease,Type 1
- A Study of AT2101 in Patients With Gaucher Disease
ClinicalTrials.gov Identifier: NCT00446550 - Recruiting
Gaucher Disease, Type 1
- International Collaborative Gaucher Group (ICGG) Gaucher Registry
ClinicalTrials.gov Identifier: NCT00358943 - Recruiting
Gaucher Disease; Cerebroside Lipidosis Syndrome; Glucocerebrosidase Deficiency Disease; Glucosylceramide Beta-Glucosidase Deficiency Disease
- Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease
ClinicalTrials.gov Identifier: NCT00244582 - Completed
Type 2 Gaucher Disease
- OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients with Neuronopathic Gaucher Disease
ClinicalTrials.gov Identifier: NCT00041535 - Active, not recruiting
Gaucher Disease
- A Study to Evaluate and Characterize the Effect of Pharmacological Chemicals on Blood From Patients With Gaucher Disease
ClinicalTrials.gov Identifier: NCT00465062 - Recruiting
Gaucher Disease
- A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
ClinicalTrials.gov Identifier: NCT00376168 - Recruiting
Gaucher Disease
- Nervous System Degeneration in Glycosphingolipid Storage Disorders
ClinicalTrials.gov Identifier: NCT00029965 - Recruiting
Gangliosidoses; Gaucher Disease
- Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations
ClinicalTrials.gov Identifier: NCT00302146 - Recruiting
Glucocerebrosidase Mutations; Gaucher Disease
- Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
ClinicalTrials.gov Identifier: NCT00351156 - Completed
Gaucher Disease
- Study on the Prognosis and Effect of Anti-Diabetic Drugs on Type-2 Diabetes Mellitus With Coronary Artery Disease
ClinicalTrials.gov Identifier: NCT00513630 - Active, not recruiting
Type 2 Diabetes; Coronary Artery Disease
- Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
ClinicalTrials.gov Identifier: NCT00004293 - Recruiting
Gaucher's Disease
- Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients with Late-Onset Pompe Disease.
ClinicalTrials.gov Identifier: NCT00158600 - Active, not recruiting
Pompe Disease (Late-onset); Glycogen storage disease type II (GSD-II); Acid Maltase Deficiency Disease; Glycogenosis 2
- Markers and Mechanisms of Vascular Disease in Type II Diabetes
ClinicalTrials.gov Identifier: NCT00256646 - Active, not recruiting
Type 2 Diabetes Mellitus
- Blood Pressure and Glucose Lowering for the Prevention of Vascular Disease in High Risk Patients with Type 2 Diabetes.
ClinicalTrials.gov Identifier: NCT00145925 - Active, not recruiting
Diabetes Mellitus, Type 2
- Post-Marketing Safety Study of GSK Biological''s Pediarix™ Vaccine.
ClinicalTrials.gov Identifier: NCT00146835 - Active, not recruiting
Prophylaxis: Diphtheria, tetanus, pertussis, hepatitis B, poliovirus type 1, type 2 and type 3
- Efficacy and Safety In Poorly Controlled Type 2 Diabetics
ClinicalTrials.gov Identifier: NCT00044460 - Active, not recruiting
type 2 diabetes
- Japanese Primary prevention of atherosclerosis with Aspirin for Diabetes (JPAD) Trial
ClinicalTrials.gov Identifier: NCT00110448 - Active, not recruiting
Coronary Disease; Arteriosclerosis; Diabetes Mellitus, Type 2
- Sulodexide to Treat Type 2 Diabetic Nephropathy
ClinicalTrials.gov Identifier: NCT00342238 - Recruiting
Overt Type 2 Diabetic Nephropathy; Chronic Kidney Disease; Diabetes Type 2
- Miglustat in Niemann-Pick Type C Disease
ClinicalTrials.gov Identifier: NCT00517153 - Active, not recruiting
Niemann-Pick Type C Disease
- An Observational Study Evaluating SYMLIN® (Pramlintide Acetate) Injection Use in Insulin Using Patients With Type 2 and Type 1 Diabetes
ClinicalTrials.gov Identifier: NCT00229658 - Active, not recruiting
Type 1 Diabetes Mellitus; Type 2 Diabetes Mellitus
- Aliskiren Trial in Type 2 Diabetes Using Cardiovascular and Renal Disease Endpoints
ClinicalTrials.gov Identifier: NCT00549757 - Recruiting
Type 2 Diabetes Mellitus; Cardiovascular Disease
- Evaluation of ALT-2074 in Subjects With Type-2 Diabetes, Haptoglobin Type 2-2 Genotype and Coronary Artery Disease
ClinicalTrials.gov Identifier: NCT00491543 - Recruiting
Diabetes; Coronary Artery Disease
- Rasagiline 1 mg and 2 mg Added to Aricept 10 mg Daily in Patients with Mild to Moderate Alzheimer's Disease (AD)
ClinicalTrials.gov Identifier: NCT00104273 - Active, not recruiting
Dementia, Alzheimer Type
- Effects of a Mediterranean Style Diet on Vascular Health in Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00163683 - Recruiting
Type 2 Diabetes; Coronary Heart Disease
- Effect of Nuts vs. a Wheat Bran Muffin in Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00410722 - Recruiting
Type 2 Diabetes; Cardiovascular Disease; Diet Therapy
- Long-Term Study of Safety and Efficacy of WelChol® as an Additional Therapy in Patients with Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00151762 - Active, not recruiting
Type 2 Diabetes
- Islet Transplantation in Type 1 Diabetics Using the Edmonton Protocol of Steroid Free Immunosuppression
ClinicalTrials.gov Identifier: NCT00133809 - Active, not recruiting
Diabetes Mellitus, Type 1
- PEG-Glucocerebrosidase for the Treatment of Gaucher Disease
ClinicalTrials.gov Identifier: NCT00001410 - Completed
Gaucher's Disease
- Safety and Immunogenicity Study of Hib-MenCY-TT Vaccine Compared to Licensed Hib and Meningococcal Serogroup C Conjugate Vaccines, Each Administered at 2, 4, 6, and 12 to 15 Months of Age.
ClinicalTrials.gov Identifier: NCT00134719 - Active, not recruiting
Invasive bacterial disease caused by Haemophilus influenzae type b and Neisseria meningitidis serogroups C and Y.
- Safety of Inhaled Insulin With Type 1 and Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00308737 - Active, not recruiting
Diabetes Mellitus, Type I; Diabetes Mellitus, Type II
- A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
ClinicalTrials.gov Identifier: NCT00515398 - Recruiting
Pompe Disease; Glycogen Storage Disease Type II
- Olmesartan Medoxomil in Diabetes Mellitus
ClinicalTrials.gov Identifier: NCT00185159 - Active, not recruiting
Diabetes Mellitus, Type 2; Cardiovascular Disease; Kidney Disease
- Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
ClinicalTrials.gov Identifier: NCT00555555 - Recruiting
Congenital Type 3 Von Willebrand Disease
- Late-Onset Treatment Study Extension Protocol
ClinicalTrials.gov Identifier: NCT00455195 - Active, not recruiting
Pompe Disease (Late-Onset); Glycogen Storage Disease Type II (GSD-II); Glycogenesis 2; Acid Maltase Deficiency
- TRx0014 in Patients With Mild or Moderate Alzheimer's Disease
ClinicalTrials.gov Identifier: NCT00515333 - Active, not recruiting
Dementia, Alzheimer Type
- A Single-Blind, Placebo-Controlled, Dose-Ranging Trial of Oral HDV-Insulin in Patients With Type 2 Diabetes Mellitus
ClinicalTrials.gov Identifier: NCT00521378 - Active, not recruiting
Diabetes Mellitus, Type 1; Diabetes Mellitus, Type 2
- Glycemic Control and Complications in Diabetes Mellitus Type 2 (VADT)
ClinicalTrials.gov Identifier: NCT00032487 - Active, not recruiting
Type 2 diabetes mellitus
- ORIENT: Olmesartan Reducing Incidence of End Stage Renal Disease in Diabetic Nephropathy Trial
ClinicalTrials.gov Identifier: NCT00141453 - Active, not recruiting
Diabetic Nephropathy; type 2 diabeted mellitus; Proteinuria
- Effects of Vitamin B1 in Type 1 Diabetic Patients
ClinicalTrials.gov Identifier: NCT00117026 - Active, not recruiting
Diabetes Mellitus, Type 1
- Pompe Disease Registry
ClinicalTrials.gov Identifier: NCT00231400 - Recruiting
Glycogen Storage Disease Type II; Pompe Disease
- Study In People With Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00196989 - Active, not recruiting
Type 2 Diabetes Mellitus
- Effect of Metformin in Patients with Type-1 Diabetes with Inadequate Glycaemic Control by Insulin and Diet
ClinicalTrials.gov Identifier: NCT00118937 - Active, not recruiting
Diabetes Mellitus, Type 1
- Botulinum Toxin Type A (BOTOX) in the Management of Levodopa-Induced Peak-Dose Dyskinesias in Parkinson's Disease
ClinicalTrials.gov Identifier: NCT00477802 - Recruiting
Parkinson Disease
- Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene into Peripheral Blood Stem Cells for Autologous Transplantation in Patients with Type I Gaucher Disease
ClinicalTrials.gov Identifier: NCT00004294 - Completed
Gaucher's Disease
- Dietary Interventions in Type 2 Obese Diabetic Patients in the Community
ClinicalTrials.gov Identifier: NCT00520182 - Active, not recruiting
Diabetes Type 2
- Influence of Physical Training on Mitochondrial Function in Type 2 Diabetic Patients
ClinicalTrials.gov Identifier: NCT00365495 - Active, not recruiting
Type 2 Diabetes
- A Study in Type 1 Diabetic Patients With Repeated Doses of E1 in Combination With G1
ClinicalTrials.gov Identifier: NCT00239148 - Active, not recruiting
Type 1 Diabetes
- A Study in Type 2 Diabetic Patients With Repeated Doses of E1 in Combination With G1
ClinicalTrials.gov Identifier: NCT00239187 - Active, not recruiting
Type 2 Diabetes
- Open Trial of Miglitol in Type 2 Diabetic Patients Treated With Biguanide
ClinicalTrials.gov Identifier: NCT00334503 - Active, not recruiting
Type 2 Diabetes Mellitus
- 4 Weeks Treatment of Type II Diabetic Patients With BI 44847
ClinicalTrials.gov Identifier: NCT00558909 - Active, not recruiting
Diabetes Mellitus, Type 2
- Surgical Treatment of Non-Obese Type 2 Diabetic Patients With Duodenal Exclusion
ClinicalTrials.gov Identifier: NCT00566358 - Active, not recruiting
Diabetes Mellitus, Type 2
- Double-Blind Trial of Miglitol in Type 2 Diabetic Patients Treated With Biguanide
ClinicalTrials.gov Identifier: NCT00334399 - Active, not recruiting
Type 2 Diabetes Mellitus
- Safety and Efficacy of PEG-Encapsulated Islet Allografts Implanted in Type I Diabetic Recipients
ClinicalTrials.gov Identifier: NCT00260234 - Active, not recruiting
Diabetes Mellitus, Type 1
- Islet Transplantation in Type 1 Diabetic Patients Using the Edmonton Protocol of Steroid Free Immunosuppression
ClinicalTrials.gov Identifier: NCT00566813 - Active, not recruiting
Diabetes Mellitus, Type 1
- Family Management of Type 1 Diabetes in Children
ClinicalTrials.gov Identifier: NCT00273286 - Active, not recruiting
Diabetes Mellitus, Type 1
- Sitagliptin vs Glipizide in Patients With Type 2 Diabetes Mellitus and End-Stage Renal Disease
ClinicalTrials.gov Identifier: NCT00509236 - Recruiting
Type 2 Diabetes Mellitus; Renal Disease, End-Stage
- Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)
ClinicalTrials.gov Identifier: NCT00258778 - Completed
Gaucher Disease
- Lifestyle Intervention in a General Population for Prevention of Ischaemic Heart Disease
ClinicalTrials.gov Identifier: NCT00289237 - Active, not recruiting
CHD; AMI; CVD; Type 2 Diabetes; COLD
- A 2 Year Random Control Trial of Patients with Type 2 Diabetes Focusing on Cardiovascular Diagnostics and Metabolic Control
ClinicalTrials.gov Identifier: NCT00133718 - Active, not recruiting
Diabetes Mellitus; Type 2 Diabetes Mellitus
- Effect of Repaglinide Versus Metformin Treatment in Non-Obese Patients With Type-2-Diabetes
ClinicalTrials.gov Identifier: NCT00118963 - Active, not recruiting
Diabetes Mellitus, Type 2
- Efficacy and Safety of Vildagliptin in Patients with Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00102388 - Active, not recruiting
Diabetes Mellitus, Type 2
- Efficacy and Safety of Vildagliptin in Patients with Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00106340 - Active, not recruiting
Diabetes Mellitus, Type 2
- Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
ClinicalTrials.gov Identifier: NCT00344331 - Recruiting
Neimann-Pick Disease
- New Onset of Type 1 Diabetes Mycophenolate Mofetil-Daclizumab Clinical Trial
ClinicalTrials.gov Identifier: NCT00100178 - Active, not recruiting
Diabetes Mellitus, Type 1
- Prevalence of Macrovascular Disease in Type 2 Diabetes Mellitus
ClinicalTrials.gov Identifier: NCT00298844 - Recruiting
Type 2 Diabetes Mellitus
- Diet, Obesity and Genes (DiOGenes)
ClinicalTrials.gov Identifier: NCT00390637 - Active, not recruiting
Obesity; Metabolic Syndrome; Type 2 Diabetes; Cardiovascular Disease
- Long-term Study of Starlix+Diovan to Delay Type II Diabetes Mellitus
ClinicalTrials.gov Identifier: NCT00097786 - Active, not recruiting
Diabetes Mellitus, Type 2
- Insulin NovoRapid Vs. Actrapid in Treatment of Type 1 Diabetic Patients During Daily Adjustment of Insulin Dose
ClinicalTrials.gov Identifier: NCT00145353 - Active, not recruiting
Type 1 diabetes mellitus
- Pilot Study of B-Type Natrieutic Peptide (BNP) Levels in Patients With Congenital Heart Disease(BNP)
ClinicalTrials.gov Identifier: NCT00308230 - Recruiting
Congenital Heart Disease; Cardiovascular Disease
- A Randomized, Controlled Study on Ca Blocker Vs. AII Antagonists in Hypertension with Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00144144 - Active, not recruiting
Hypertension; Diabetes Mellitus, Type 2
- The DURABLE Trial: Evaluating the Durability of Starter Insulin Regimens in Patients With Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00279201 - Active, not recruiting
Diabetes Mellitus, Type 2
- Safety of Insulin Detemir Produced by a New Process as Measured by Antibody Formation in Type 1 Diabetes
ClinicalTrials.gov Identifier: NCT00447382 - Active, not recruiting
Diabetes Mellitus, Type 1
- Effect of Long Term α-Lipoic Acid Treatment on Endothelial Function in Patients With Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00490867 - Active, not recruiting
Type 2 Diabetes
- Outcomes and Cost Consequences of Using an Internet Co-Management Module to Improve the Quality of Type 1 Diabetes Care
ClinicalTrials.gov Identifier: NCT00194558 - Active, not recruiting
Diabetes Mellitus, Type 1
- Study of Weight Loss Using Gastric Stimulation in Obese Patients With Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00200018 - Active, not recruiting
Severe to Morbid Obesity and Type 2 Diabetes
- Placebo-Controlled Study of the Combination of SYR-322 and Pioglitazone HCl in the Treatment of Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00328627 - Active, not recruiting
Type 2 Diabetes Mellitus
- Efficacy of Vildagliptin 100 MG as Compared to Placebo as Add-on to Metformin in Patients With Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00351884 - Active, not recruiting
Diabetes Mellitus, Type 2
- Effect of Liraglutide in Combination With Sulfonylurea (SU) on Blood Glucose Control in Subjects With Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00395746 - Active, not recruiting
Diabetes Mellitus, Type 2
- Natural History Study of Plexiform Neurofibromas in Patients With Neurofibromatosis Type 1
ClinicalTrials.gov Identifier: NCT00445991 - Active, not recruiting
Neurofibromatosis Type 1 (nf1); Precancerous/Nonmalignant Condition
- Safety and Efficacy of Inhaled Pre-Prandial Human Insulin in Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00343980 - Active, not recruiting
Diabetes Mellitus, Type 2
- Randomized Study of Real-Time Continuous Glucose Monitors in the Management of Type 1 Diabetes
ClinicalTrials.gov Identifier: NCT00406133 - Active, not recruiting
Type 1 Diabetes
- Impact of Self-Monitoring Blood Glucose Frequency on Glycemic Control in Patients With Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00202033 - Active, not recruiting
Type 2 Diabetes
- A Study Assessing Saxagliptin Treatment in Subjects With Type 2 Diabetes Who Are Not Controlled With Diet and Exercise
ClinicalTrials.gov Identifier: NCT00374907 - Active, not recruiting
Type 2 Diabetes
- Drug Interactions From Simultaneous Administration Of Metformin And GSK189075 To Subjects With Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00376038 - Active, not recruiting
Type 2 Diabetes Mellitus
- Long Term Safety of Vildagliptin (100mg qd) In Patients With Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00389415 - Active, not recruiting
Diabetes Mellitus, Type 2
- A Titration Study of a GLP-1 Analogue in Patients With Type 2 Diabetes Treated With Metformin.
ClinicalTrials.gov Identifier: NCT00460941 - Active, not recruiting
Diabetes Mellitus, Type 2
- Effect of Real Time Continuous Glucose Monitoring System on the Management of Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00536549 - Active, not recruiting
Type 2 Diabetes
- Duration of The Honeymoon Phase of Type 1 Diabetes: A Comparison of Insulins Detemir, Glargine and NPH
ClinicalTrials.gov Identifier: NCT00564018 - Enrolling by invitation
Type 1 Diabetes
- Efficacy and Safety of Vildagliptin Compared to Placebo on the Incretin Effect in Patients With Type 2 Diabetes Treated With Metformin
ClinicalTrials.gov Identifier: NCT00396071 - Active, not recruiting
Diabetes Mellitus, Type 2
- Effect of Liraglutide or Exenatide Added to an Ongoing Treatment on Blood Glucose Control in Subjects With Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00518882 - Active, not recruiting
Type 2 Diabetes Mellitus
- A Concordance Study Comparing Insulin Treatments in Patients With Type 2 Diabetes
ClinicalTrials.gov Identifier: NCT00330473 - Active, not recruiting
Type 2 Diabetes Mellitus

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